Cereno Scientific Reports Positive Phase IIa Results for CS1 in Pulmonary Arterial Hypertension

Cereno Scientific, a leading biotech firm listed on Nasdaq First North, has reported positive topline results from its Phase IIa clinical trial for the drug CS1, aimed at treating Pulmonary Arterial Hypertension (PAH). PAH is a severe, life-threatening condition characterized by elevated blood pressure in the pulmonary arteries, leading to heart failure and death. Existing treatments inadequately address the root causes of the disease, underpinning the necessity for novel therapeutic approaches.

The recent trial demonstrated that CS1 is safe and well-tolerated in patients, meeting the primary endpoint. In addition to safety, the drug showed a positive impact on various clinical parameters important to both patients and regulatory authorities. These parameters include risk scores, functional class, and hemodynamics such as mean Pulmonary Arterial Pressure (mPAP) and Pulmonary Vascular Resistance (PVR). CEO Sten R. Sörensen expressed his enthusiasm, highlighting the trial results as a significant advancement in developing CS1 as a disease-modifying therapy for PAH.

PAH, a rare and fatal disease, is driven by epigenetic mechanisms involving histone deacetylase (HDAC) that lead to continuous deterioration of pulmonary artery function. The disease results in endothelial dysfunction, inflammation, vasoconstriction, and hypertrophy, all contributing to poor lung function and right heart failure. Current treatments primarily focus on alleviating symptoms rather than addressing the underlying causes, making CS1's disease-modifying potential particularly promising.

Dr. Raymond Benza, the Network Director of Pulmonary Hypertension at Mount Sinai Icahn School of Medicine and Principal Investigator of the CS1 Phase IIa trial, emphasized the need for effective, safe, and disease-modifying therapies for PAH patients. He noted that the trial results, which include improvements in risk scores, functional class, and mean pulmonary pressure, are encouraging.

CS1 is an HDAC inhibitor developed to reverse the pathological vascular remodeling of small lung arteries, aiming to be a disease-modifying treatment for PAH. The CS1-003 Phase IIa trial evaluated the safety, tolerability, pharmacokinetics, and exploratory efficacy of CS1 in 25 PAH patients. The trial, conducted at ten clinical sites in the US, confirmed that CS1 is well-tolerated and has a positive impact on clinical efficacy parameters over a 12-week period.

Dr. Jason Guichard, an investigator in the trial, reported positive outcomes in patients treated with CS1, noting improvements in their health and well-being. The data from the trial, combined with preclinical information, suggests CS1 has the potential to reverse pathological remodeling in PAH, a critical factor in disease progression.

Cereno Scientific aims to initiate discussions with regulatory bodies to pursue a pivotal trial for CS1, buoyed by the encouraging results. The company will conduct a comprehensive analysis of the complete CS1-003 data and present the findings at upcoming medical conferences and in reputable medical journals. Dr. Rahul Agrawal, CMO and Head of R&D at Cereno Scientific, expressed optimism about the future development of CS1 and its potential to make a meaningful impact on PAH patients' lives.

The CS1-003 trial, titled “A Phase II, Prospective, Randomized, Open-label, Blinded Endpoint, Multicenter Study,” involved adult PAH patients with WHO Functional Class II or III. These patients were stable on standard therapy for 90 days and were randomized to one of three daily doses of CS1. The trial's primary endpoint was to assess the safety and tolerability of CS1, alongside exploratory evaluations of other standard endpoints.

Pulmonary Arterial Hypertension affects approximately 10 in 100,000 people globally. It is characterized by progressive narrowing of the pulmonary arterial vessels, which can lead to right heart failure and death. Life expectancy for PAH patients is significantly shortened, with current treatments offering only moderate improvements.

CS1, as an HDAC inhibitor, works through epigenetic modulation, showing potential as a safe, effective, and disease-modifying treatment for PAH. The drug has demonstrated a unique efficacy profile, aligning well with the underlying mechanisms of PAH. CS1 aims to improve the quality of life and extend the survival of PAH patients, addressing a critical unmet need in current treatment options.

Since January 2024, CS1 has been approved by the FDA for Expanded Access, allowing patients who have completed the Phase IIa trial to continue treatment under a formal protocol. This initiative supports both the treatment of PAH patients and the collection of long-term safety and efficacy data, aiding future regulatory discussions and the planning of Phase IIb/III pivotal studies.

Cereno Scientific continues to develop innovative treatments for cardiovascular diseases, with CS1 leading its pipeline of HDAC inhibitors. The company is headquartered in Gothenburg, Sweden, with a subsidiary in Boston, Massachusetts, and is listed on Nasdaq First North.

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